TY - JOUR
T1 - Extracutaneous involvement is common and associated with prolonged disease activity and greater impact in juvenile localized scleroderma
AU - Li, Suzanne C.
AU - Higgins, Gloria C.
AU - Chen, Mallory
AU - Torok, Kathryn S.
AU - Rabinovich, C. Egla
AU - Stewart, Katie
AU - Laxer, Ronald M.
AU - Pope, Elena
AU - Haines, Kathleen A.
AU - Punaro, Marilynn
AU - O'Neil, Kathleen M.
N1 - Funding Information:
This work was supported by an Arthritis Foundation New Jersey Chapter Grant. Additional support for meetings and calls was provided by the Childhood Arthritis and Rheumatology Research Alliance (CARRA).
Publisher Copyright:
© 2021 The Author(s) 2021. Published by Oxford University Press on behalf of the British Society for Rheumatology. All rights reserved. For permissions, please email: journals.permissions@oup.com.
PY - 2021/12/1
Y1 - 2021/12/1
N2 - Objective: The aim of this study was to evaluate factors associated with extracutaneous involvement (ECI) in juvenile localized scleroderma (jLS). Methods: A prospective, multicentre, 6-month observational study was performed. The data collected included disease features, global assessments, and subject symptoms. Bivariate and linear multilevel regression analyses were performed. Results: A total of 86 jLS subjects (80% female, 80% Caucasian), median age of disease onset 7.7 years, were evaluated. Most had linear scleroderma or mixed morphea. Of the 86 subjects, 49 (57%) had 125 extracutaneous problems {median 2 [interquartile range (IQR) 1, 3] per subject} from nine organ systems. Most of these subjects had multiple musculoskeletal problems. ECI was associated with more extensive cutaneous involvement, higher number of symptoms, family history of autoimmunity, and ANA and RF positivity. Subjects with ECI had higher scores for physician global assessment of damage (PGA-D), and parental global assessment of disease impact, but not baseline physician global assessment of disease activity (PGA-A). Although subjects with ECI received more MTX and glucocorticoid treatment, they had a slower reduction in PGA-A scores and symptoms over time, suggesting a poorer response to treatment. In logistic regression modelling, female sex had the largest effect on parental impact scores. Conclusion: ECI occurred in the majority of subjects with jLS, and was associated with more medication use, longer treatment duration, higher PGA-D scores, and higher parental assessment of disease impact. Our findings suggest that jLS subjects with ECI have greater overall disease burden, both cutaneous and extracutaneous, and poorer response to treatment. More study of the treatment needs of this population is warranted.
AB - Objective: The aim of this study was to evaluate factors associated with extracutaneous involvement (ECI) in juvenile localized scleroderma (jLS). Methods: A prospective, multicentre, 6-month observational study was performed. The data collected included disease features, global assessments, and subject symptoms. Bivariate and linear multilevel regression analyses were performed. Results: A total of 86 jLS subjects (80% female, 80% Caucasian), median age of disease onset 7.7 years, were evaluated. Most had linear scleroderma or mixed morphea. Of the 86 subjects, 49 (57%) had 125 extracutaneous problems {median 2 [interquartile range (IQR) 1, 3] per subject} from nine organ systems. Most of these subjects had multiple musculoskeletal problems. ECI was associated with more extensive cutaneous involvement, higher number of symptoms, family history of autoimmunity, and ANA and RF positivity. Subjects with ECI had higher scores for physician global assessment of damage (PGA-D), and parental global assessment of disease impact, but not baseline physician global assessment of disease activity (PGA-A). Although subjects with ECI received more MTX and glucocorticoid treatment, they had a slower reduction in PGA-A scores and symptoms over time, suggesting a poorer response to treatment. In logistic regression modelling, female sex had the largest effect on parental impact scores. Conclusion: ECI occurred in the majority of subjects with jLS, and was associated with more medication use, longer treatment duration, higher PGA-D scores, and higher parental assessment of disease impact. Our findings suggest that jLS subjects with ECI have greater overall disease burden, both cutaneous and extracutaneous, and poorer response to treatment. More study of the treatment needs of this population is warranted.
KW - damage
KW - extracutaneous manifestation
KW - linear scleroderma
KW - morbidity
KW - morphea
KW - symptoms
KW - treatment response
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U2 - 10.1093/rheumatology/keab238
DO - 10.1093/rheumatology/keab238
M3 - Article
C2 - 33711155
AN - SCOPUS:85114180730
SN - 1462-0324
VL - 60
SP - 5724
EP - 5733
JO - Rheumatology (United Kingdom)
JF - Rheumatology (United Kingdom)
IS - 12
ER -